Tuesday, May 17, 2016

Globe Put This in Your CAR-T

Announcing the birth of a new biotech company tends to follow a well-worn path: There’s a breathless press release explaining the technology, a list of moneyed investors buying into it, and a sizable dollar figure thrown in for gravitas.

But a new oncology upstart is turning heads in the industry with none of those things. Instead, Vor BioPharma is getting off the ground with few details on its unproven approach to cancer and enlisting the aid of a scientist whose big prize is named for Joseph Pulitzer, not Alfred Nobel.

What’s more, the Boston-based company is wading into a field crowded with competition, where “if they’re going to go me-too, they’re going to lose,” said Eric Schmidt, a biotech analyst at Cowen Group.

Vor is working on technology that uses the immune system to fight cancer. The process entails removing patients’ disease-fighting T cells and reengineering them to home in on the telltale signs of tumors. The resulting products, called CAR-T cells, are then injected back into the body as newly armed tumor-killers.

CAR-T isn’t terribly new. Novartis, with help from the University of Pennsylvania, has demonstrated stellar clinical results with the technology in lymphoma and plans to submit its lead therapy for marketing approval next year. And behind the Swiss drug giant are a group of smaller US firms including Juno Therapeutics and Kite Pharma, all of which have successfully treated human tumors with their technology and are moving toward regulatory filings.

But Vor, launched on Monday by Boston startup factory PureTech Health, is years behind. Its CAR-T candidates are still being studied on mice, and the company has no timeline for clinical trials.

Furthermore, Vor’s bedrock science comes from a surprising source: Dr. Siddhartha Mukherjee, a Columbia University physician-scientist who won the Pulitzer Prize in 2011 for his book “The Emperor of All Maladies: A Biography of Cancer.”

As oncologists go, Mukherjee is about as famous as it gets. His last book was adapted into a PBS miniseries by the awarding-winning documentarian Ken Burns, and his latest one, “The Gene: An Intimate History,” has been well-received.

But his resume as a drug hunter is comparatively light. Though Mukherjee has long worked in blood cancer, and he has a couple dozen journal articles to his name, his research has never crossed over into drug development. He has never published an academic paper on CAR-T, and his only listed patent is unrelated to the field.

Mukherjee isn’t the only scientific mind at work within Vor, though. The company’s Acting Chief Scientific Officer is Joseph Bolen, who filled the same role for 14 years at Millennium Pharmaceuticals, where he developed the blockbuster cancer drug Velcade. Joining him as scientific founders are Stanford University radiologist Dr. Sanjiv Sam Gambhir, New York University immunologist Dr. Dan Littman, and Harvard University stem cell biologist Derrick Rossi, all of whom have advised academic spinoffs in the past.

What brought them all together, Rossi said, was the promise of Mukherjee’s CAR-T discoveries. And Rossi believes Vor’s work will eventually clear up any skepticism around Mukherjee’s bona fides in the field.

“What’s published in a lab and what’s not published in a lab — the actual work going on — are two completely different things,” Rossi told STAT. “There’s a lot of really exciting science coming that hasn’t seen the light of publication yet.”

One thing Vor is disclosing: The young company is looking for areas where its bigger and better-established forbears haven’t succeeded. To date, the majority of CAR-T projects have focused on leukemias and lymphomas that stem from white blood cells called B cells, but according to PureTech associate Aleks Radovic-Moreno, Vor is staying out of that field altogether, training its efforts on cancers outside the realm of B cells.

It’s early days for Vor, and it will take years to determine whether the company can eventually compete with the likes of Novartis, Juno, and Kite. But cancer is “a massive opportunity with tons of unmet need,” Cowen’s Schmidt said, and there’s plenty of room for new ideas. “Just because there are other well-funded CAR-T companies ahead of them doesn’t mean they couldn’t have success.”

That's rather chilling, and given the overwhelming lobbying power of pharmaceuticals, quite frightening going forward.Why would you want to kill a cash cow?

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